CU24574B1 - Oligonucleótido que contiene una secuencia nucleobase para disminuir la expresión de tau - Google Patents

Oligonucleótido que contiene una secuencia nucleobase para disminuir la expresión de tau

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Publication number
CU24574B1
CU24574B1 CU2018000065A CU20180065A CU24574B1 CU 24574 B1 CU24574 B1 CU 24574B1 CU 2018000065 A CU2018000065 A CU 2018000065A CU 20180065 A CU20180065 A CU 20180065A CU 24574 B1 CU24574 B1 CU 24574B1
Authority
CU
Cuba
Prior art keywords
nucleobase sequence
oligonucleotide containing
tau expression
decrease
oligonucleotides
Prior art date
Application number
CU2018000065A
Other languages
English (en)
Spanish (es)
Other versions
CU20180065A7 (es
Inventor
Ofengeim Manuela Polydoro
Jan Weiler
Original Assignee
Novartis Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of CU20180065A7 publication Critical patent/CU20180065A7/es
Publication of CU24574B1 publication Critical patent/CU24574B1/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2320/00—Applications; Uses
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CU2018000065A 2015-12-21 2016-12-19 Oligonucleótido que contiene una secuencia nucleobase para disminuir la expresión de tau CU24574B1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562270165P 2015-12-21 2015-12-21
PCT/IB2016/057794 WO2017109679A1 (fr) 2015-12-21 2016-12-19 Compositions et méthodes pour diminier l'expression de tau

Publications (2)

Publication Number Publication Date
CU20180065A7 CU20180065A7 (es) 2018-11-06
CU24574B1 true CU24574B1 (es) 2022-01-13

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Family Applications (1)

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CU2018000065A CU24574B1 (es) 2015-12-21 2016-12-19 Oligonucleótido que contiene una secuencia nucleobase para disminuir la expresión de tau

Country Status (40)

Country Link
US (2) US11473083B2 (fr)
EP (3) EP3394259B1 (fr)
JP (4) JP7027328B2 (fr)
KR (2) KR20250112919A (fr)
CN (2) CN116218848A (fr)
AR (1) AR107579A1 (fr)
AU (2) AU2016376034B2 (fr)
CA (1) CA3008094A1 (fr)
CL (3) CL2018001652A1 (fr)
CO (1) CO2018006297A2 (fr)
CR (1) CR20200609A (fr)
CU (1) CU24574B1 (fr)
DK (1) DK3394259T3 (fr)
DO (1) DOP2018000151A (fr)
EA (2) EA036417B1 (fr)
EC (1) ECSP18050575A (fr)
ES (1) ES2984631T3 (fr)
FI (1) FI3394259T3 (fr)
HR (1) HRP20240672T1 (fr)
HU (1) HUE066548T2 (fr)
IL (3) IL305866B2 (fr)
JO (2) JO3790B1 (fr)
LT (1) LT3394259T (fr)
MA (2) MA71468A (fr)
MX (2) MX2018007726A (fr)
MY (1) MY191737A (fr)
PE (2) PE20181448A1 (fr)
PH (1) PH12018501207B1 (fr)
PL (1) PL3394259T3 (fr)
PT (1) PT3394259T (fr)
RS (1) RS65535B1 (fr)
RU (1) RU2747734C2 (fr)
SA (1) SA518391831B1 (fr)
SG (2) SG11201804704PA (fr)
SI (1) SI3394259T1 (fr)
SV (1) SV2018005714A (fr)
TW (2) TWI787163B (fr)
UY (1) UY37038A (fr)
WO (1) WO2017109679A1 (fr)
ZA (1) ZA201803661B (fr)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2831232A4 (fr) 2012-03-30 2015-11-04 Univ Washington Procédés de modulation de l'expression de tau pour réduire l'avc et modifier un symptôme neurodégénératif
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
HUE065170T2 (hu) 2016-09-29 2024-05-28 Biogen Ma Inc Vegyületek és módszerek a TAU expresszió csökkentésére
MX2020009532A (es) * 2018-03-13 2020-10-05 Janssen Pharmaceutica Nv Oligonucleotidos modificados para uso en el tratamiento de tauopatias.
PE20210346A1 (es) 2018-07-03 2021-02-25 Hoffmann La Roche Oligonucleotidos para modular la expresion de tau
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof
CN113817728A (zh) * 2020-06-19 2021-12-21 四川大学华西医院 一种有效干扰Tau蛋白表达的重组慢病毒及其应用
AU2022293669A1 (en) * 2021-06-16 2024-01-18 Empirico Inc. Treatment of mtres1 related diseases and disorders
EP4370680A4 (fr) * 2021-07-16 2025-08-13 Aptah Bio Inc Compositions polynucléotidiques et procédés de régulation de l'expression génique
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
EP4441224A4 (fr) * 2021-12-03 2026-03-11 Quralis Corp Oligonucléotides antisens gapmères avec des produits chimiques de squelette modifiés
EP4608981A1 (fr) * 2022-10-25 2025-09-03 Empirico Inc. Traitement par thérapie génique de maladies et de troubles associés à mtres1
WO2024155740A1 (fr) * 2023-01-18 2024-07-25 Aptah Bio, Inc. Compositions polynucléotidiques et méthodes de traitement du cancer
CN121127586A (zh) * 2023-01-18 2025-12-12 阿普塔生物公司 用于治疗神经退行性疾病的多核苷酸组合物和方法
TW202448484A (zh) * 2023-04-20 2024-12-16 美商雅迪克斯製藥公司 Ｍａｐｔ調節組合物及其使用方法
US20250283084A1 (en) * 2024-03-08 2025-09-11 Eli Lilly And Company Mapt antisense oligonucleotide
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026025082A1 (fr) * 2024-07-25 2026-01-29 Biogen Ma Inc. Méthodes de traitement de la maladie d'alzheimer

Family Cites Families (106)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
KR927003044A (ko) 1990-01-11 1992-12-17 크리스토퍼 케이. 미라벨리 Rna 활성과 유전자 발현을 검출하고 조절하는 조성물 및 그 방법
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020052331A1 (en) 1990-08-14 2002-05-02 Brenda Baker Compositions and methods for antisense inhibition of protein translation
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
DK0618968T3 (da) 1991-12-06 2000-04-10 Max Planck Gesellschaft Midler til diagnostisering og behandling af Alzheimer's sygdom
US7408027B1 (en) 1991-12-06 2008-08-05 Max-Planck-Gesellschaft Zur Forderung Der Wissenchaften Tools for the diagnosis and treatment of Alzheimer's disease
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
NZ256974A (en) * 1992-10-05 1997-12-19 Isis Pharmaceuticals Inc Oligonucleotides and compositions thereof for modulating expression of the human ras gene
KR940021073A (ko) 1993-03-02 1994-10-17 미야베 요시가즈 알쯔하이머병의 예방 또는 치료제, 그 스크리닝 방법 및 인간 유래의 타우 단백질 키나아제 i
CA2116460A1 (fr) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Agents prophylactiques ou therapeutiques pour la maladie d'alzheimer, methode de depistage de la maladie d'alzheimer et tau-proteine kinase i extraite d'un organisme humain
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
NZ331217A (en) 1996-02-14 2000-02-28 Novartis Ag Sugar-modified gapped oligonucleotides for eliciting RNase H activity for strand cleavage in an opposing strand
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
WO1999057129A1 (fr) 1998-05-01 1999-11-11 Mayo Foundation For Medical Education And Research Mutations tau pathogenes
US6664443B1 (en) 1998-05-01 2003-12-16 Mayo Foundation For Medical Education And Research Pathogenic Tau mutations in transgenic mice
HUP0203035A3 (en) * 1998-07-14 2007-12-28 Corixa Corp Compositions and methods for therapy and diagnosis of prostate cancer
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
WO2000064262A1 (fr) 1999-04-26 2000-11-02 The University Of North Carolina At Chapel Hill Sequences de fucosyltransferase humaines anti-sens et methodes d'utilisation correspondantes
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
CN101284873A (zh) * 1999-06-30 2008-10-15 科里克萨有限公司 用于肺癌治疗和诊断的组合物和方法
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
AU2001229619A1 (en) 2000-01-21 2001-07-31 Pharmacia And Upjohn Company Transgenic mouse model of human neurodegenerative disease
AU2001241839A1 (en) * 2000-02-28 2001-09-12 Decode Genetics Ehf Human schizophrenia gene
WO2002055681A2 (fr) 2001-01-11 2002-07-18 Bayer Ag Regulation de la tau-tubuline kinase humaine
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
WO2004003134A2 (fr) 2002-06-26 2004-01-08 Isis Pharmaceuticals, Inc. Inhibition antisens via une reduction independante de la ribonuclease h dans un arnm
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
JP4393382B2 (ja) 2002-08-14 2010-01-06 三菱化学株式会社 中枢性タウ蛋白質特異的抗体
WO2004041889A2 (fr) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Composes oligomeres renfermant un substitut de sucre polycyclique et compositions intervenant dans la modulation genique
US8163896B1 (en) 2002-11-14 2012-04-24 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7906326B2 (en) 2003-05-07 2011-03-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides associated with alzheimer's disease and uses thereof
US20060257851A1 (en) 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20070077553A1 (en) 2003-10-30 2007-04-05 Rosetta Genomics Bioinformatically detectable group of novel vaccinia regulatory genes and uses thereof
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US20130130231A1 (en) 2002-11-26 2013-05-23 Isaac Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US7696334B1 (en) 2002-12-05 2010-04-13 Rosetta Genomics, Ltd. Bioinformatically detectable human herpesvirus 5 regulatory gene
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
EP1660657A1 (fr) 2003-08-28 2006-05-31 Novartis AG Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'
EP1683862B1 (fr) * 2003-09-25 2012-08-01 Chiba-Prefecture Microreseau d'evaluation de pronostic neuroblastome et procede d'evaluation de pronostic de neuroblastome
US20070106066A1 (en) 2003-10-28 2007-05-10 Alexander Cherkasky Cherkasky fusion proteins containing antibody-, antigen- and microtubule-binding regions and immune response-triggering regions
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
JP5697297B2 (ja) * 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
EP2933332A1 (fr) 2004-06-28 2015-10-21 The University Of Western Australia Oligonucléotides antisens permettant d'induire un saut d'exon et leurs procédés d'utilisation
US7312035B2 (en) 2004-09-03 2007-12-25 Affymetrix, Inc. Methods of genetic analysis of yeast
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
JP5921052B2 (ja) 2004-12-03 2016-05-24 ロード アイランド ホスピタル アルツハイマー病の検査
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (fr) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions et procédés pour la modulation de l'expression du gène lmna
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
EP2007888A2 (fr) 2006-04-03 2008-12-31 Santaris Pharma A/S Composition pharmaceutique comprenant des oligonucleotides antisens anti-miarn
WO2008002620A2 (fr) 2006-06-27 2008-01-03 University Of Virginia Patent Foundation Modèle cellulaire pour pathologie de la maladie d'alzheimer
AU2007323469B2 (en) 2006-11-23 2014-07-24 Guangdong Maijinjia Biotechnology Co., Ltd Oligonucleotides for modulating target RNA activity
EP2145014B1 (fr) 2007-04-05 2012-12-12 The J. David Gladstone Institutes Agents de reduction de surexcitation neuronale
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
CA2717792A1 (fr) 2008-03-07 2009-09-11 Santaris Pharma A/S Compositions pharmaceutiques pour le traitement de maladies associees aux microarn
ES2599979T3 (es) 2009-04-24 2017-02-06 Roche Innovation Center Copenhagen A/S Composiciones farmacéuticas para el tratamiento de pacientes de VHC que no responden al interferón
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
WO2011094645A1 (fr) 2010-01-28 2011-08-04 University Of Massachusetts Lowell Méthodes de diagnostic de maladies neurodégénératives associées à tau
WO2011117353A1 (fr) 2010-03-24 2011-09-29 Mirrx Therapeutics A/S Oligonucléotides antisens bivalents
CA2817256A1 (fr) 2010-11-12 2012-05-18 The General Hospital Corporation Arn non codants associes a polycomb
WO2012069059A1 (fr) 2010-11-23 2012-05-31 Mirrx Therapeutics A/S Oligonucléotides pour la modulation de l'activité d'arn cibles
EP2655621B1 (fr) 2010-12-20 2018-05-23 The General Hospital Corporation Arn non codants associés à polycomb
EP3495497B1 (fr) * 2011-04-28 2021-03-24 Life Technologies Corporation Procédés et compositions pour pcr multiplexe
WO2013013165A2 (fr) 2011-07-21 2013-01-24 The Trustees Of The University Of Pennsylvania Utilisation de composés d'acide nucléique bloqué (lna) pour l'inhibition thérapeutique de mir-21 dans le lupus érythémateux systémique
EP2831232A4 (fr) * 2012-03-30 2015-11-04 Univ Washington Procédés de modulation de l'expression de tau pour réduire l'avc et modifier un symptôme neurodégénératif
WO2013151665A2 (fr) 2012-04-02 2013-10-10 modeRNA Therapeutics Polynucléotides modifiés destinés à la production de protéines associées à une maladie humaine
EP2855520B1 (fr) 2012-06-04 2018-09-26 Novartis AG Méthodes de marquage spécifiques à un site et molécules ainsi produites
JP6243919B2 (ja) 2012-10-24 2017-12-06 フラウンホーファ−ゲゼルシャフト ツァー フォルデルング デア アンゲバンデン フォルシュンク エー． ファオ．Ｆｒａｕｎｈｏｆｅｒ−Ｇｅｓｅｌｌｓｃｈａｆｔ Ｚｕｒ Ｆｏｒｄｅｒｕｎｇ Ｄｅｒ Ａｎｇｅｗａｎｄｔｅｎ Ｆｏｒｓｃｈｕｎｇ Ｅ． Ｖ． 微小管修飾化合物
US9200068B2 (en) 2012-12-18 2015-12-01 Regents Of The University Of Minnesota Compositions and methods related to tauopathy
HK1216902A1 (zh) 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140315977A1 (en) 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CN105264091B (zh) * 2013-03-14 2020-02-28 Ionis制药公司 用于调节tau表达的组合物和方法
EP3007705A4 (fr) 2013-06-12 2017-02-15 Oncoimmunin, Inc. Administration systémique in vivo d'oligonucléotides
TW202246503A (zh) * 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2015068075A2 (fr) 2013-10-24 2015-05-14 Institut National De La Sante Et De La Recherche Medicale Détection de protéine tau
CN104711324A (zh) * 2013-12-11 2015-06-17 芮屈生物技术（上海）有限公司 老年性痴呆病变前期TAU基因的mRNA水平原位杂交筛查试剂盒
EP3736334A1 (fr) * 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Compositions d'arni pour le traitement de pathologies liées à l'apoc3
WO2016019063A1 (fr) 2014-07-29 2016-02-04 Isis Pharmaceuticals, Inc. Compositions et méthodes pour moduler l'expression de tau
WO2016127002A1 (fr) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Oligonucléotides lna à flancs alternés
CN115181778A (zh) 2015-02-04 2022-10-14 百时美施贵宝公司 选择治疗性分子的方法
US11077132B2 (en) * 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases

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